Exondys Approval: Measured Efficacy Outcomes Vs. Patient 'Anecdotes'
Executive Summary
Pink Sheet’s Drug Review Profile looks at US FDA clinical memos opposing approval of Sarepta's Duchenne muscular dystrophy drug eteplirsen, revealing challenges patient advocates and agency face in transforming patient experiences into hard data that can be used for approvals.
You may also be interested in...
Drug Review Profiles: Behind The Scenes At US FDA
A year in our long-running series of deep dives into FDA review documents.
US FDA's Search For Meaning: Interpretation Of Regulatory Language Can Inspire Conflict In Drug Reviews
The Pink Sheet's Drug Review Profile series illustrates the challenge of reconciling review realities and broad statutory directives.
Before Price Controversy, Emflaza Got Easy US Nod With Old But Good Enough Data
Pink Sheet's monthly Drug Review Profile looks at Marathon's (now PTC's) Emflaza for Duchenne muscular dystrophy, and finds that before the steroid's price ignited controversy it received an unremarkable review – unremarkable, that is, for rare and serious illnesses. The US FDA accepted old and flawed trial data because signals of clinical benefit were consistently seen and safety profile was understood.